Just 6 months after springing into existing with a pivotal-stage program in the clinic under an experienced set of executives and investigators, biotech spinoff Spark Therapeutics has gathered a whopping $72.8 million venture round designed to get through a Phase III study and well down the road to its goal of becoming a fully integrated company.
Sofinnova Ventures led this latest round for Spark, with additional cash coming from Brookside Capital, Deerfield Management, Rock Springs Capital, funds and accounts managed by T. Rowe Price Associates, Wellington Management and two undisclosed funds. Children's Hospital of Philadelphia (CHOP), which co-founded the company with tech it developed, also played a big role in the round.
Their money will be used to advance a late-stage trial for a new gene therapy that addresses RPE65-related retinal dystrophies. Work on its gene therapies had begun at CHOP's Center for Cellular and Molecular Therapeutics, which launched about a decade ago. A group of people who were involved in the work took positions at Spark with plans to build a full slate of clinical, regulatory and manufacturing expertise as some prominent investigators--including Jean Bennett--pursue roles as scientific advisers to the newly created biotech. Its Series A round came in at $50 million.
Just a couple of weeks ago Spark outlined plans to build a 28,000-square-foot facility with access to manufacturing capacity and a blueprint to quickly build the staff to 50.
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| Spark Therapeutics CEO Jeffrey Marrazzo |
"With our combination of industry-leading gene therapy expertise, deep pipeline, and strong clinical results to-date, we've been able to attract a diverse set of blue-chip investors, giving us capital to maintain our position as a leader in the gene therapy field," said Jeffrey Marrazzo, co-founder and CEO of Spark Therapeutics, in a statement. "The funding will support the expansion of our team and ongoing development of our pipeline as we build the infrastructure needed for a first-in-class, FDA-approved gene therapy."
This new round, part of which was drawn from the originally announced Series A, funds the company out for a full three years. By next year the biotech believes it will have Phase III data in hand that can be taken to the FDA. Behind that is a therapy now in a clinical trial for hemophilia B, with another program that is waiting in the wings that follows on its lead therapy.
Now's the time to expand on the core group at Spark with outside hires who can help develop a full set of talents that will be needed to continue to accelerate the growth of the company, Marrazzo tells FierceBiotech. And the CEO is confident that Spark and indeed the whole field is on the cusp of "some very nice successes."
"What we've seen is that many of the initial challenges, while not all solved, many of them have solutions," says Marrazzo. "This is an evolution from research and scientific development to new product development. That's the phase we're in."
Spark isn't closing the door to partnerships. Marrazzo says the biotech will do collaborations with other companies when they can help move along broader programs at a faster pace. But he's particularly keen on inking more tie-ups like the recent pact Spark signed with Genable, an Irish biotech that wants to tap Spark's expertise in exploring a two-vector technology, helping expand on Spark's knowledge and capabilities in rare eye diseases.
Bennett, by the way, was the co-author of a recent micro study that used gene therapy to treat a handful of patients with Leber congenital amaurosis, which leads to blindness. Bennett used an adeno-associated virus to transport a corrective gene into the eyes of three adult patients. Bennett said the patients responded well, demonstrating improved vision. And significantly, the treatment was completed without any harmful immune response, which has raised questions in the past about the safety of gene therapy.
- here's the release (PDF)